IL245674B - Methods and preparations for the targeted modification of a genome - Google Patents

Methods and preparations for the targeted modification of a genome

Info

Publication number
IL245674B
IL245674B IL245674A IL24567416A IL245674B IL 245674 B IL245674 B IL 245674B IL 245674 A IL245674 A IL 245674A IL 24567416 A IL24567416 A IL 24567416A IL 245674 B IL245674 B IL 245674B
Authority
IL
Israel
Prior art keywords
genome
compositions
methods
targeted modification
targeted
Prior art date
Application number
IL245674A
Other languages
English (en)
Hebrew (he)
Other versions
IL245674A0 (en
Original Assignee
Regeneron Pharma
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of IL245674A0 publication Critical patent/IL245674A0/en
Publication of IL245674B publication Critical patent/IL245674B/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276Knock-out vertebrates
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • C12N15/1024In vivo mutagenesis using high mutation rate "mutator" host strains by inserting genetic material, e.g. encoding an error prone polymerase, disrupting a gene for mismatch repair
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/035Animal model for multifactorial diseases
    • A01K2267/0362Animal model for lipid/glucose metabolism, e.g. obesity, type-2 diabetes
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/035Animal model for multifactorial diseases
    • A01K2267/0387Animal model for diseases of the immune system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8527Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/40Systems of functionally co-operating vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/10Vectors comprising a non-peptidic targeting moiety
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/40Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2999/00Further aspects of viruses or vectors not covered by groups C12N2710/00 - C12N2796/00 or C12N2800/00
    • C12N2999/007Technological advancements, e.g. new system for producing known virus, cre-lox system for production of transgenic animals

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Environmental Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Husbandry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
IL245674A 2013-12-11 2016-05-17 Methods and preparations for the targeted modification of a genome IL245674B (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361914768P 2013-12-11 2013-12-11
US201462017416P 2014-06-26 2014-06-26
US201462029261P 2014-07-25 2014-07-25
US201462052906P 2014-09-19 2014-09-19
US201462059527P 2014-10-03 2014-10-03
US201462064384P 2014-10-15 2014-10-15
PCT/US2014/060788 WO2015088643A1 (en) 2013-12-11 2014-10-15 Methods and compositions for the targeted modification of a genome

Publications (2)

Publication Number Publication Date
IL245674A0 IL245674A0 (en) 2016-06-30
IL245674B true IL245674B (en) 2020-04-30

Family

ID=51830654

Family Applications (1)

Application Number Title Priority Date Filing Date
IL245674A IL245674B (en) 2013-12-11 2016-05-17 Methods and preparations for the targeted modification of a genome

Country Status (12)

Country Link
US (7) US9546384B2 (zh)
JP (3) JP6174811B2 (zh)
KR (2) KR102170502B1 (zh)
CN (2) CN105980568B (zh)
AU (3) AU2014360811B2 (zh)
BR (1) BR112016013400B1 (zh)
CA (1) CA2933433C (zh)
IL (1) IL245674B (zh)
MX (2) MX2016007654A (zh)
RU (2) RU2685914C1 (zh)
SG (1) SG10201700961TA (zh)
WO (1) WO2015088643A1 (zh)

Families Citing this family (183)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US9242014B2 (en) 2010-06-15 2016-01-26 The Regents Of The University Of California Receptor tyrosine kinase-like orphan receptor 1 (ROR1) single chain Fv antibody fragment conjugates and methods of use thereof
US9217040B2 (en) 2011-01-14 2015-12-22 The Regents Of The University Of California Therapeutic antibodies against ROR-1 protein and methods for use of same
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
WO2013163394A1 (en) 2012-04-25 2013-10-31 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors
PT2800811T (pt) 2012-05-25 2017-08-17 Univ California Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição
EP2888283B1 (en) 2012-08-24 2018-09-19 The Regents of The University of California Antibodies and vaccines for use in treating ror1 cancers and inhibiting metastasis
ES2769310T3 (es) 2012-12-06 2020-06-25 Sigma Aldrich Co Llc Modificación y regulación del genoma basada en CRISPR
RU2690352C2 (ru) 2013-02-20 2019-05-31 Регенерон Фармасютикалс, Инк. Генетическая модификация крыс
CN110540991B (zh) 2013-03-15 2023-10-24 通用医疗公司 使用截短的引导RNA(tru-gRNA)提高RNA引导的基因组编辑的特异性
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
JP6411463B2 (ja) 2013-04-16 2018-10-24 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. ラットゲノムの標的改変
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015040402A1 (en) * 2013-09-18 2015-03-26 Kymab Limited Methods. cells & organisms
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
RU2685914C1 (ru) * 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
CN106133141B (zh) 2014-01-08 2021-08-20 哈佛学院董事及会员团体 Rna引导的基因驱动
EP3102722B1 (en) 2014-02-04 2020-08-26 Jumpcode Genomics, Inc. Genome fractioning
PL3105328T3 (pl) 2014-02-11 2020-10-19 The Regents Of The University Of Colorado, A Body Corporate Umożliwiana przez CRISPR multipleksowa modyfikacja genomu
MX2020004803A (es) * 2014-05-30 2021-10-13 Regeneron Pharma Animales con dipeptidil peptidasa iv (dpp4) humanizada.
PL3152312T3 (pl) 2014-06-06 2020-08-10 Regeneron Pharmaceuticals, Inc. Sposoby i kompozycje do modyfikowania docelowego locus
MX2016017317A (es) 2014-06-26 2017-08-24 Regeneron Pharma Metodos y composiciones para modificaciones geneticas dirigidas y metodos de uso.
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US10428310B2 (en) 2014-10-15 2019-10-01 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
WO2016065341A1 (en) 2014-10-24 2016-04-28 Avectas Limited Delivery across cell plasma membranes
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
WO2016081924A1 (en) * 2014-11-20 2016-05-26 Duke University Compositions, systems and methods for cell therapy
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
RU2707137C2 (ru) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации посредством одноэтапного множественного нацеливания
WO2016138292A1 (en) 2015-02-25 2016-09-01 Igenomx International Genomics Corporation Methods and compositions for in silico long read sequencing
RU2744831C2 (ru) 2015-03-16 2021-03-16 Регенерон Фармасьютикалз, Инк. Не относящееся к человеку животное, у которого проявляется снижение функции верхних и нижних моторных нейронов и чувственного восприятия
ES2905525T3 (es) 2015-05-06 2022-04-11 Snipr Tech Ltd Alteración de poblaciones microbianas y modificación de la microbiota
EP3310159A4 (en) 2015-06-16 2019-02-20 The Jackson Laboratory GENETICALLY MODIFIED NON-MENTAL ANIMALS AND METHOD FOR COMPLEMENTARY CYTOTOXICITY
WO2017004261A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
AU2016301195B2 (en) 2015-08-06 2022-09-01 Dana-Farber Cancer Institute, Inc. Targeted protein degradation to attenuate adoptive T-cell therapy associated adverse inflammatory responses
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
CA2998894A1 (en) 2015-09-18 2017-03-23 The Regents Of The University Of California Methods for autocatalytic genome editing and neutralizing autocatalytic genome editing and compositions thereof
KR102645625B1 (ko) * 2015-09-22 2024-03-07 제넨테크, 인크. Fc-함유 단백질의 발현
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
IL294014B1 (en) 2015-10-23 2024-03-01 Harvard College Nucleobase editors and their uses
JP2018532415A (ja) * 2015-11-06 2018-11-08 ザ ジャクソン ラボラトリー 大きなゲノムdnaノックインおよびその使用
WO2017096041A1 (en) * 2015-12-02 2017-06-08 The Regents Of The University Of California Compositions and methods for modifying a target nucleic acid
CA3009715A1 (en) 2015-12-30 2017-07-06 Avectas Limited Vector-free delivery of gene editing proteins and compositions to cells and tissues
CA3013932A1 (en) * 2016-02-12 2017-08-17 Jumpcode Genomics, Inc. Method for target specific rna transcription of dna sequences
US11339427B2 (en) 2016-02-12 2022-05-24 Jumpcode Genomics, Inc. Method for target specific RNA transcription of DNA sequences
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
IL299000A (en) * 2016-04-04 2023-02-01 Eth Zuerich A mammalian cell line for protein production and a breeding library
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
WO2017189336A1 (en) * 2016-04-25 2017-11-02 The Regents Of The University Of California Methods and compositions for genomic editing
EP3457840B1 (en) * 2016-05-20 2024-04-10 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
CN113907043B (zh) 2016-06-03 2023-08-01 瑞泽恩制药公司 表达外源末端脱氧核苷酸转移酶的非人动物
GB201609811D0 (en) 2016-06-05 2016-07-20 Snipr Technologies Ltd Methods, cells, systems, arrays, RNA and kits
US11293021B1 (en) 2016-06-23 2022-04-05 Inscripta, Inc. Automated cell processing methods, modules, instruments, and systems
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
US10688181B2 (en) 2016-06-27 2020-06-23 The Regents Of The University Of California Cancer treatment combinations
EP3490373B1 (en) 2016-07-29 2023-10-25 Regeneron Pharmaceuticals, Inc. Mice comprising mutations resulting in expression of c-truncated fibrillin-1
JP7184364B2 (ja) * 2016-08-02 2022-12-06 国立大学法人京都大学 ゲノム編集のための方法
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
CN106544360B (zh) * 2016-10-17 2019-11-05 扬州大学 一种终止lncRNA双等位基因转录的方法
US20190390221A1 (en) * 2016-11-02 2019-12-26 David Kiewlich Plasmid vectors for expression of large nucleic acid transgenes
DK3766343T3 (da) 2016-11-04 2022-06-20 Regeneron Pharma Ikke-humane dyr med et manipuleret immunoglobulin-lambda-letkædelocus
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
AU2018210420A1 (en) 2017-01-19 2019-08-08 Omniab, Inc. Human antibodies from transgenic rodents with multiple heavy chain immunoglobulin loci
JP7227912B2 (ja) 2017-02-08 2023-02-24 ダナ-ファーバー キャンサー インスティテュート,インコーポレイテッド キメラ抗原受容体の調節
EP3583201B1 (en) * 2017-02-14 2024-01-17 University of Pittsburgh - Of the Commonwealth System of Higher Education Methods of engineering human induced pluripotent stem cells to produce liver tissue
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN106987604B (zh) * 2017-03-29 2021-05-28 北京希诺谷生物科技有限公司 一种制备动脉粥样硬化疾病模型犬的方法
EP3612551A4 (en) 2017-04-21 2020-12-30 The General Hospital Corporation VARIANTS OF CPF1 (CAS12A) WITH MODIFIED PAM SPECIFICITY
WO2018197520A1 (en) * 2017-04-24 2018-11-01 Dupont Nutrition Biosciences Aps Methods and compositions of anti-crispr proteins for use in plants
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP2020521446A (ja) 2017-05-25 2020-07-27 ザ ジェネラル ホスピタル コーポレイション 二部塩基エディター(bbe)構造およびii型−c−cas9ジンクフィンガー編集
CN109136273B (zh) * 2017-06-16 2022-09-27 中国科学院分子细胞科学卓越创新中心 制备免疫缺陷的大鼠的方法及其应用
RU2020100919A (ru) * 2017-06-20 2021-07-20 Цзянсу Хэнжуй Медсин Ко., Лтд. СПОСОБ НОКАУТИРОВАНИЯ ГЕНА-МИШЕНИ В T-КЛЕТКЕ IN VITRO И crRNA, ПРИМЕНЯЕМАЯ В ДАННОМ СПОСОБЕ
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
US11696572B2 (en) 2017-06-27 2023-07-11 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ASGR1 locus
HRP20220615T1 (hr) 2017-06-30 2022-06-24 Inscripta, Inc. Postupci, moduli, instrumenti i sustavi za automatiziranu obradu stanica
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
WO2019018534A1 (en) 2017-07-18 2019-01-24 The Board Of Trustees Of Leland Stanford Junior University GENOME EDITION WITHOUT SCARCE THROUGH TWO-STEP APPROVED REPAIR BY HOMOLOGY
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
JP2020533957A (ja) 2017-07-31 2020-11-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. Crisprリポーター非ヒト動物およびその使用
BR112019027673A2 (pt) 2017-07-31 2020-09-15 Regeneron Pharmaceuticals, Inc. animal não humano, e, métodos para testar a recombinação induzida por crispr/cas e para otimizar a capacidade de crispr/cas
KR20200032693A (ko) 2017-07-31 2020-03-26 리제너론 파마슈티칼스 인코포레이티드 Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도
US20190045761A1 (en) * 2017-08-11 2019-02-14 Recombinetics, Inc. Inducible disease models methods of making them and use in tissue complementation
US10738327B2 (en) 2017-08-28 2020-08-11 Inscripta, Inc. Electroporation cuvettes for automation
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
EP3679143A1 (en) * 2017-09-08 2020-07-15 Life Technologies Corporation Methods for improved homologous recombination and compositions thereof
AU2018338790B2 (en) 2017-09-29 2022-09-15 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TTR locus and methods of use
CA3074927A1 (en) 2017-09-30 2019-04-04 Inscripta, Inc. Flow through electroporation instrumentation
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
KR102444458B1 (ko) 2017-11-10 2022-09-19 리제너론 파마슈티칼스 인코포레이티드 Slc30a8 돌연변이를 포함하는 비인간 동물 및 사용 방법
SG11202002456WA (en) * 2017-11-30 2020-04-29 Regeneron Pharma Non-human animals comprising a humanized trkb locus
US11643670B2 (en) 2018-01-29 2023-05-09 Massachusetts Institute Of Technology Methods of enhancing chromosomal homologous recombination
KR102647714B1 (ko) 2018-03-19 2024-03-18 리제너론 파마슈티칼스 인코포레이티드 CRISPR/Cas 시스템을 사용한 동물에서의 전사 조절
US10760075B2 (en) 2018-04-30 2020-09-01 Snipr Biome Aps Treating and preventing microbial infections
AU2019242586A1 (en) 2018-03-26 2020-10-01 Regeneron Pharmaceuticals, Inc. Humanized rodents for testing therapeutic agents
CN112204131A (zh) 2018-03-29 2021-01-08 因思科瑞普特公司 用于诱导和转化的细胞生长速率的自动化控制
US10376889B1 (en) 2018-04-13 2019-08-13 Inscripta, Inc. Automated cell processing instruments comprising reagent cartridges
US10557216B2 (en) 2018-04-24 2020-02-11 Inscripta, Inc. Automated instrumentation for production of T-cell receptor peptide libraries
US10858761B2 (en) 2018-04-24 2020-12-08 Inscripta, Inc. Nucleic acid-guided editing of exogenous polynucleotides in heterologous cells
WO2019209926A1 (en) 2018-04-24 2019-10-31 Inscripta, Inc. Automated instrumentation for production of peptide libraries
EP3794130A4 (en) 2018-05-16 2022-07-27 Synthego Corporation METHODS AND SYSTEMS FOR DESIGN AND USE OF GUIDE RNA
US11142740B2 (en) 2018-08-14 2021-10-12 Inscripta, Inc. Detection of nuclease edited sequences in automated modules and instruments
US10532324B1 (en) 2018-08-14 2020-01-14 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
US10752874B2 (en) 2018-08-14 2020-08-25 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
IL292273B2 (en) 2018-08-14 2023-10-01 Inscripta Inc Devices, modules and methods for improved detection of edited sequences in living cells
EP3844263A4 (en) 2018-08-30 2022-05-25 Inscripta, Inc. ENHANCED DETECTION OF NUCLEASE-EDITED SEQUENCES IN AUTOMATED MODULES AND INSTRUMENTS
WO2020056122A1 (en) 2018-09-13 2020-03-19 Regeneron Pharmaceuticals, Inc. Complement factor h gene knockout rat as a model of c3 glomerulopathy
US11851663B2 (en) 2018-10-14 2023-12-26 Snipr Biome Aps Single-vector type I vectors
WO2020086475A1 (en) 2018-10-22 2020-04-30 Inscripta, Inc. Engineered enzymes
US11214781B2 (en) 2018-10-22 2022-01-04 Inscripta, Inc. Engineered enzyme
US11965172B2 (en) 2018-11-05 2024-04-23 California Institute Of Technology DNA sequence modification-based gene drive
CN111321171A (zh) * 2018-12-14 2020-06-23 江苏集萃药康生物科技有限公司 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法
JP7449291B2 (ja) 2018-12-20 2024-03-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヌクレアーゼ媒介リピート伸長
JP7144618B2 (ja) * 2018-12-20 2022-09-29 北京大学 バーコード付きガイドrna構築体を使用する効率的な遺伝子スクリーニングのための組成物及び方法
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
WO2020168315A1 (en) 2019-02-15 2020-08-20 Just-Evotec Biologics, Inc. Automated biomanufacturing systems, facilities, and processes
NL2022714B1 (en) * 2019-03-11 2020-09-18 Academisch Ziekenhuis Leiden Optimised RAG1 deficient SCID Gene Therapy
EP3942040A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US11001831B2 (en) 2019-03-25 2021-05-11 Inscripta, Inc. Simultaneous multiplex genome editing in yeast
CN113631713A (zh) 2019-03-25 2021-11-09 因思科瑞普特公司 酵母中的同时多重基因组编辑
SG11202108454RA (en) 2019-04-04 2021-09-29 Regeneron Pharma Non-human animals comprising a humanized coagulation factor 12 locus
CN113966401A (zh) * 2019-04-10 2022-01-21 犹他大学研究基金会 用于治疗amd的htra1调节
EP3978607A4 (en) * 2019-05-27 2023-01-25 Transgenic Inc. HUMANIZED EXON MOUSE
KR20220016869A (ko) 2019-06-04 2022-02-10 리제너론 파마슈티칼스 인코포레이티드 베타-슬립 돌연변이를 갖는 인간화 ttr 좌위를 포함하는 비-인간 동물 및 사용 방법
CA3139122C (en) 2019-06-06 2023-04-25 Inscripta, Inc. Curing for recursive nucleic acid-guided cell editing
MX2021015122A (es) 2019-06-07 2022-04-06 Regeneron Pharma Animales no humanos que comprenden un locus de albumina humanizado.
US10907125B2 (en) 2019-06-20 2021-02-02 Inscripta, Inc. Flow through electroporation modules and instrumentation
CN114008070A (zh) 2019-06-21 2022-02-01 因思科瑞普特公司 导致大肠杆菌赖氨酸产量增加的全基因组合理设计的突变
US10927385B2 (en) 2019-06-25 2021-02-23 Inscripta, Inc. Increased nucleic-acid guided cell editing in yeast
WO2021102059A1 (en) 2019-11-19 2021-05-27 Inscripta, Inc. Methods for increasing observed editing in bacteria
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021118626A1 (en) 2019-12-10 2021-06-17 Inscripta, Inc. Novel mad nucleases
US10704033B1 (en) 2019-12-13 2020-07-07 Inscripta, Inc. Nucleic acid-guided nucleases
KR20220118498A (ko) 2019-12-18 2022-08-25 인스크립타 인코포레이티드 핵산-가이드된 뉴클레아제 편집된 세포의 생체 내 검출을 위한 캐스케이드/dcas3 상보 검정
US10689669B1 (en) 2020-01-11 2020-06-23 Inscripta, Inc. Automated multi-module cell processing methods, instruments, and systems
AU2021213705A1 (en) 2020-01-27 2022-06-16 Inscripta, Inc. Electroporation modules and instrumentation
US20210227812A1 (en) 2020-01-28 2021-07-29 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized pnpla3 locus and methods of use
EP4099821A1 (en) 2020-02-07 2022-12-14 Regeneron Pharmaceuticals, Inc. <smallcaps/>? ? ?klkb1? ? ? ? ?non-human animals comprising a humanizedlocus and methods of use
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
EP4134431A4 (en) * 2020-04-06 2024-06-05 Logomix, Inc. GENOME ALTERATION METHOD AND GENOME ALTERATION KIT
US20210332388A1 (en) 2020-04-24 2021-10-28 Inscripta, Inc. Compositions, methods, modules and instruments for automated nucleic acid-guided nuclease editing in mammalian cells
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
US11787841B2 (en) 2020-05-19 2023-10-17 Inscripta, Inc. Rationally-designed mutations to the thrA gene for enhanced lysine production in E. coli
BR112022024652B1 (pt) * 2020-06-06 2024-01-16 Lanzatech, Inc Micro-organismo geneticamente modificado, e, método para a produção de um produto
CN111647663B (zh) * 2020-06-18 2022-07-01 安徽农业大学 一种鸡步行数性状的分子遗传标记及应用
WO2021263146A2 (en) 2020-06-26 2021-12-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
WO2022060749A1 (en) 2020-09-15 2022-03-24 Inscripta, Inc. Crispr editing to embed nucleic acid landing pads into genomes of live cells
US11512297B2 (en) 2020-11-09 2022-11-29 Inscripta, Inc. Affinity tag for recombination protein recruitment
EP4271802A1 (en) 2021-01-04 2023-11-08 Inscripta, Inc. Mad nucleases
WO2022150269A1 (en) 2021-01-07 2022-07-14 Inscripta, Inc. Mad nucleases
WO2022154343A1 (ko) * 2021-01-13 2022-07-21 재단법인대구경북과학기술원 Apoe christchurch 돌연변이를 포함하는 알츠하이머 저항성 세포 모델 및 이의 제조방법
JP2024505524A (ja) 2021-01-29 2024-02-06 メルク・シャープ・アンド・ドーム・エルエルシー プログラム死受容体1(pd-1)抗体の組成物及びその組成物を得る方法
US11884924B2 (en) 2021-02-16 2024-01-30 Inscripta, Inc. Dual strand nucleic acid-guided nickase editing
CA3212642A1 (en) 2021-03-05 2022-09-09 The Board Of Trustees Of The Leland Stanford Junior University In vivo dna assembly and analysis
EP4370647A1 (en) 2021-07-15 2024-05-22 Just-Evotec Biologics, Inc. Bidirectional tangential flow filtration (tff) perfusion system
CN118251491A (zh) 2021-10-28 2024-06-25 瑞泽恩制药公司 用于敲除C5的CRISPR/Cas相关方法及组合物
AU2022381205A1 (en) 2021-11-04 2024-03-28 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified cacng1 locus
CA3238939A1 (en) 2021-12-08 2023-06-15 Gaurang Patel Mutant myocilin disease model and uses thereof
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023122506A1 (en) 2021-12-20 2023-06-29 Regeneron Pharmaceuticals, Inc. Non-human animals comprising humanized ace2 and tmprss loci
WO2023150798A1 (en) 2022-02-07 2023-08-10 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
WO2023154861A1 (en) 2022-02-11 2023-08-17 Regeneron Pharmaceuticals, Inc. Compositions and methods for screening 4r tau targeting agents
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2024026488A2 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified transferrin receptor locus
WO2024031053A1 (en) 2022-08-05 2024-02-08 Regeneron Pharmaceuticals, Inc. Aggregation-resistant variants of tdp-43
WO2024073679A1 (en) 2022-09-29 2024-04-04 Regeneron Pharmaceuticals, Inc. Correction of hepatosteatosis in humanized liver animals through restoration of il6/il6r/gp130 signaling in human hepatocytes

Family Cites Families (224)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5981175A (en) 1993-01-07 1999-11-09 Genpharm Internation, Inc. Methods for producing recombinant mammalian cells harboring a yeast artificial chromosome
DE19525285C2 (de) 1995-06-28 1999-04-15 Inst Pflanzengenetik & Kultur In vitro Testverfahren zum Nachweis Chemikalien-induzierter embryotoxischer/teratogener Effekte
EP0880584A1 (en) 1996-02-16 1998-12-02 University Of Edinburgh Cytokine expressed by dia/lif-deficient embryonic stem cells for the inhibition of differentiation
US5830729A (en) 1996-04-18 1998-11-03 Institut Pasteur I Sce I-induced gene replacement and gene conversion in embryonic stem cells
US6136566A (en) 1996-10-04 2000-10-24 Lexicon Graphics Incorporated Indexed library of cells containing genomic modifications and methods of making and utilizing the same
AU8587598A (en) 1997-07-26 1999-02-16 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
WO2000039316A1 (en) 1998-12-31 2000-07-06 The J. David Gladstone Institutes Transgenic rodents and rodent cell lines expressing hiv co-receptors
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
AU2982900A (en) 1999-02-03 2000-08-25 Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
US20030104526A1 (en) 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
ATE309536T1 (de) 1999-12-06 2005-11-15 Sangamo Biosciences Inc Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
AU2884102A (en) 2000-12-07 2002-06-18 Sangamo Biosciences Inc Regulation of angiogenesis with zinc finger proteins
CA2435394C (en) 2001-01-22 2018-01-09 Sangamo Biosciences, Inc. Modified zinc finger binding proteins
AU2002225187A1 (en) 2001-01-22 2002-07-30 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
AU2003251286B2 (en) 2002-01-23 2007-08-16 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20060078552A1 (en) 2002-03-15 2006-04-13 Sylvain Arnould Hybrid and single chain meganucleases and use thereof
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
CA2497913C (en) 2002-09-05 2014-06-03 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
AU2003290518A1 (en) 2002-09-06 2004-04-23 Fred Hutchinson Cancer Research Center Methods and compositions concerning designed highly-specific nucleic acid binding proteins
US20030175968A1 (en) 2002-10-30 2003-09-18 Golic Kent G. Gene targeting method
US7344886B2 (en) 2002-11-29 2008-03-18 Boehringer Ingelheim Pharma Gmbh & Co., Kg Neomycin-phosphotransferase-genes and methods for the selection of recombinant cells producing high levels of a desired gene product
AU2004204509A1 (en) 2003-01-13 2004-07-29 Mario R. Capecchi Persistent expression of candidate molecule in proliferating stem and progenitor cells for delivery of therapeutic products
WO2005001087A2 (en) 2003-06-11 2005-01-06 Regeneron Pharmaceuticals, Inc. Methods of modifying genes in eukaryotic cells
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
JP4862119B2 (ja) 2004-03-04 2012-01-25 大日本住友製薬株式会社 ラット胚性幹細胞
EP1591521A1 (en) 2004-04-30 2005-11-02 Cellectis I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof
WO2006028723A1 (en) 2004-09-03 2006-03-16 Moraga Biotechnology Inc. Non-embryonic totipotent blastomer-like stem cells and methods therefor
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
DK2767161T3 (en) 2004-10-19 2018-05-07 Regeneron Pharma Method of generating an animal homozygous for genetic modification
FR2879622B1 (fr) 2004-12-17 2008-02-01 Agronomique Inst Nat Rech Procede in vitro de production d'ovocytes ou d'oeufs presentant une modification genomique ciblee
AU2006224248B2 (en) 2005-03-15 2011-01-06 Cellectis I-Crei meganuclease variants with modified specificity, method of preparation and uses thereof
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
US10022457B2 (en) 2005-08-05 2018-07-17 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
GB0615327D0 (en) 2006-03-30 2006-09-13 Univ Edinburgh Culture medium containing kinase inhibitors and uses thereof
AU2007232393A1 (en) 2006-03-30 2007-10-11 The University Court Of The University Of Edinburgh Culture medium containing kinase inhibitors. and uses thereof
WO2007117410A2 (en) 2006-03-31 2007-10-18 Medarex, Inc. Transgenic animals expressing chimeric antibodies for use in preparing human antibodies
CN101117633B (zh) 2006-08-03 2011-07-20 上海交通大学附属儿童医院 一种细胞核移植方法
JP5632610B2 (ja) 2006-12-14 2014-11-26 ダウ アグロサイエンシィズ エルエルシー 最適化された非正準ジンクフィンガータンパク質
US7771967B2 (en) 2006-12-22 2010-08-10 The J. David Gladstone Institutes Nucleic acid encoding apolipoprotein E-I3
US10155038B2 (en) 2007-02-02 2018-12-18 Yale University Cells prepared by transient transfection and methods of use thereof
WO2008133938A2 (en) 2007-04-26 2008-11-06 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
CN101784664B (zh) 2007-06-01 2013-04-10 Omt公司 用于抑制内源性免疫球蛋白基因和生产转基因人独特型抗体的组合物和方法
AU2008335178A1 (en) 2007-12-10 2009-06-18 Ablexis Llc Methods for sequential replacement of targeted region by homologous recombination
KR101759586B1 (ko) 2008-08-22 2017-07-19 상가모 테라퓨틱스, 인코포레이티드 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
EP2180058A1 (en) 2008-10-23 2010-04-28 Cellectis Meganuclease recombination system
KR101803737B1 (ko) 2008-12-04 2017-12-01 상가모 테라퓨틱스, 인코포레이티드 징크 핑거 뉴클레아제를 이용한 랫트의 유전체 편집
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
JP5932632B2 (ja) 2009-03-20 2016-06-15 サンガモ バイオサイエンシーズ, インコーポレイテッド 改変された亜鉛フィンガータンパク質を使用したcxcr4の修飾
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
US20120204278A1 (en) 2009-07-08 2012-08-09 Kymab Limited Animal models and therapeutic molecules
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
US8518392B2 (en) 2009-08-14 2013-08-27 Regeneron Pharmaceuticals, Inc. Promoter-regulated differentiation-dependent self-deleting cassette
US10913929B2 (en) 2009-10-13 2021-02-09 Stemcell Technologies Inc. Method of differentiating stem cells
WO2011051390A1 (en) 2009-10-28 2011-05-05 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Homologous recombination in the oocyte
CA2779858C (en) 2009-10-29 2019-10-29 Aris N. Economides Multifunctional alleles
US20120315670A1 (en) 2009-11-02 2012-12-13 Gen9, Inc. Compositions and Methods for the Regulation of Multiple Genes of Interest in a Cell
JP5807862B2 (ja) 2009-12-01 2015-11-10 国立研究開発法人国立がん研究センター ラット胚性幹細胞を用いたキメララットの作製法
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
JP5924773B2 (ja) 2009-12-21 2016-05-25 キージーン・エン・フェー プロトプラストを形質移入するための改善された技術
SG181690A1 (en) 2009-12-21 2012-07-30 Regeneron Pharma Humanized fcy r mice
NZ600546A (en) 2010-01-22 2014-08-29 Dow Agrosciences Llc Excision of transgenes in genetically modified organisms
US20130045492A1 (en) 2010-02-08 2013-02-21 Regeneron Pharmaceuticals, Inc. Methods For Making Fully Human Bispecific Antibodies Using A Common Light Chain
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
AU2011256838B2 (en) 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
GB201009732D0 (en) 2010-06-10 2010-07-21 Gene Bridges Gmbh Direct cloning
CA2800534C (en) 2010-06-11 2019-01-22 Regeneron Pharmaceuticals, Inc. Production of fertile xy female animals from xy es cells
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
WO2012018726A1 (en) 2010-08-02 2012-02-09 Cellectis Sa Method for increasing double-strand break-induced gene targeting
US9528124B2 (en) 2013-08-27 2016-12-27 Recombinetics, Inc. Efficient non-meiotic allele introgression
WO2012129198A1 (en) 2011-03-23 2012-09-27 Transposagen Biopharmaceuticals, Inc. Genetically modified rat models for obesity and diabetes
WO2012162561A2 (en) 2011-05-24 2012-11-29 Zyngenia, Inc. Multivalent and monovalent multispecific complexes and their uses
WO2012168307A2 (en) 2011-06-07 2012-12-13 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Improved recombination efficiency by inhibition of nhej dna repair
AU2012327204B2 (en) 2011-10-28 2016-01-21 Regeneron Pharmaceuticals, Inc. Genetically modified T cell receptor mice
GB2496375A (en) 2011-10-28 2013-05-15 Kymab Ltd A non-human assay vertebrate comprising human antibody loci and human epitope knock-in, and uses thereof
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
WO2013163394A1 (en) 2012-04-25 2013-10-31 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors
CN104471067B (zh) 2012-05-07 2020-08-14 桑格摩生物治疗股份有限公司 用于核酸酶介导的转基因靶向整合的方法和组合物
PT2800811T (pt) 2012-05-25 2017-08-17 Univ California Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição
WO2013188522A2 (en) 2012-06-12 2013-12-19 Genentech, Inc. Methods and compositions for generating conditional knock-out alleles
EP3494997B1 (en) 2012-07-25 2019-09-18 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
CN110643600A (zh) 2012-10-23 2020-01-03 基因工具股份有限公司 用于切割靶dna的系统及其用途
ES2769310T3 (es) 2012-12-06 2020-06-25 Sigma Aldrich Co Llc Modificación y regulación del genoma basada en CRISPR
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
EP4279588A3 (en) 2012-12-12 2024-01-17 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
MX2015007550A (es) 2012-12-12 2017-02-02 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas.
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2931892B1 (en) 2012-12-12 2018-09-12 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP3825401A1 (en) 2012-12-12 2021-05-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
SG10201912327SA (en) 2012-12-12 2020-02-27 Broad Inst Inc Engineering and Optimization of Improved Systems, Methods and Enzyme Compositions for Sequence Manipulation
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP3553174A1 (en) 2012-12-17 2019-10-16 President and Fellows of Harvard College Rna-guided human genome engineering
JP6583918B2 (ja) 2012-12-27 2019-10-02 キージーン ナムローゼ フェンノートシャップ 植物における遺伝連鎖を解消するための方法
WO2014127287A1 (en) 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
RU2690352C2 (ru) 2013-02-20 2019-05-31 Регенерон Фармасютикалс, Инк. Генетическая модификация крыс
WO2014130955A1 (en) 2013-02-25 2014-08-28 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
JP2016507244A (ja) 2013-02-27 2016-03-10 ヘルムホルツ・ツェントルム・ミュンヒェン・ドイチェス・フォルシュンクスツェントルム・フューア・ゲズントハイト・ウント・ウムベルト(ゲーエムベーハー)Helmholtz Zentrum MuenchenDeutsches Forschungszentrum fuer Gesundheit und Umwelt (GmbH) Cas9ヌクレアーゼによる卵母細胞における遺伝子編集
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
ES2901396T3 (es) 2013-03-14 2022-03-22 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
EP2975942B1 (en) 2013-03-21 2018-08-08 Sangamo Therapeutics, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2014172458A1 (en) 2013-04-16 2014-10-23 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
JP6411463B2 (ja) * 2013-04-16 2018-10-24 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. ラットゲノムの標的改変
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
EP2796558A1 (en) 2013-04-23 2014-10-29 Rheinische Friedrich-Wilhelms-Universität Bonn Improved gene targeting and nucleic acid carrier molecule, in particular for use in plants
US10920192B2 (en) 2013-04-23 2021-02-16 Yeda Research And Development Co. Ltd. Isolated naive pluripotent stem cells and methods of generating same
CA2910427C (en) 2013-05-10 2024-02-20 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
US9890393B2 (en) 2013-05-29 2018-02-13 Cellectis Methods for engineering T cells for immunotherapy by using RNA-guided CAS nuclease system
ES2883131T3 (es) 2013-05-29 2021-12-07 Cellectis Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN
JP7065564B2 (ja) 2013-05-29 2022-05-12 セレクティス Cas9ニッカーゼ活性を用いて正確なdna切断をもたらすための方法
US20140359795A1 (en) 2013-05-31 2014-12-04 Recombinetics, Inc. Genetic techniques for making animals with sortable sperm
WO2014201015A2 (en) 2013-06-11 2014-12-18 The Regents Of The University Of California Methods and compositions for target dna modification
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
EP3011034B1 (en) 2013-06-17 2019-08-07 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
AU2014281026B2 (en) 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
WO2014204728A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
EP3011011A4 (en) 2013-06-19 2017-05-31 Sigma-Aldrich Co. LLC Targeted integration
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ CRISPR/Cas系を用いるゲノム編集の治療用の使用
RU2764637C2 (ru) 2013-07-09 2022-01-19 Президент Энд Фэллоуз Оф Харвард Коллидж Мультиплексная геномная инженерия, направляемая рнк
BR112016000571B1 (pt) 2013-07-10 2023-12-26 President And Fellows Of Harvard College Métodos in vitro para modular a expressão e para alterar um ou mais ácidos nucleicos alvo em uma célula simultaneamente com a regulação da expressão de um ou mais ácidos nucleicos alvo em uma célula, bem como célula de levedura ou bactéria compreendendo ácidos nucleicos
US9663782B2 (en) 2013-07-19 2017-05-30 Larix Bioscience Llc Methods and compositions for producing double allele knock outs
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
MX2016002118A (es) 2013-08-22 2016-06-28 Du Pont Modificacion del genoma de plantas por medio del uso de sistemas de ácido ribonucléico (arn) guía/ endonucleasa cas y metodos de uso.
US9925248B2 (en) 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015040402A1 (en) 2013-09-18 2015-03-26 Kymab Limited Methods. cells & organisms
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
CN105899665B (zh) 2013-10-17 2019-10-22 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
WO2015068785A1 (ja) 2013-11-06 2015-05-14 国立大学法人広島大学 核酸挿入用ベクター
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US10450586B2 (en) 2013-11-28 2019-10-22 Horizon Discovery Limited Somatic haploid human cell line
RU2685914C1 (ru) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
CN105899657A (zh) 2013-12-12 2016-08-24 布罗德研究所有限公司 用于改变基因产物表达的crispr-cas系统和方法、结构信息以及诱导型模块化cas酶
KR20160089530A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Hbv 및 바이러스 질병 및 질환을 위한 crispr­cas 시스템 및 조성물의 전달,용도 및 치료적 적용
MX2016007328A (es) 2013-12-12 2017-07-19 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
CN106536729A (zh) 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
JP6542226B2 (ja) 2013-12-13 2019-07-10 セレクティス ヌクレアーゼを使用して藻類形質転換細胞を選択する新しい方法
US9476065B2 (en) 2013-12-19 2016-10-25 Amyris, Inc. Methods for genomic integration
CN106133141B (zh) 2014-01-08 2021-08-20 哈佛学院董事及会员团体 Rna引导的基因驱动
JP6479024B2 (ja) 2014-01-24 2019-03-06 ザ チルドレンズ メディカル センター コーポレーション 抗体親和性の最適化のための高スループットマウスモデル
WO2015117041A1 (en) 2014-01-30 2015-08-06 Nair Ramesh B Gene modification-mediated methods and compositions for generating dominant traits in eukaryotic systems
US10233456B2 (en) 2014-01-30 2019-03-19 The Board Of Trustees Of The University Of Arkansas Method, vectors, cells, seeds and kits for stacking genes into a single genomic site
PL3105328T3 (pl) 2014-02-11 2020-10-19 The Regents Of The University Of Colorado, A Body Corporate Umożliwiana przez CRISPR multipleksowa modyfikacja genomu
US10370680B2 (en) 2014-02-24 2019-08-06 Sangamo Therapeutics, Inc. Method of treating factor IX deficiency using nuclease-mediated targeted integration
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
ES2821149T3 (es) 2014-03-12 2021-04-23 Prec Biosciences Inc Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente
RS65213B1 (sr) 2014-03-14 2024-03-29 Cibus Us Llc Postupci i kompozicije za povećanje efikasnosti ciljane modifikacije gena korišćenjem reparacije gena posredovane oligonukleotidima
US9624498B2 (en) 2014-03-18 2017-04-18 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
EP3122877B1 (en) 2014-03-26 2023-03-22 University of Maryland, College Park Targeted genome editing in zygotes of domestic large animals
GB201406968D0 (en) 2014-04-17 2014-06-04 Green Biologics Ltd Deletion mutants
KR101823661B1 (ko) 2014-04-24 2018-01-30 기초과학연구원 마이크로 상동 기반의 유전자 녹아웃을 위한 뉴클레아제 표적 서열을 확인하는 방법
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
WO2015173436A1 (en) 2014-05-16 2015-11-19 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
WO2015184268A1 (en) 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
PL3152312T3 (pl) 2014-06-06 2020-08-10 Regeneron Pharmaceuticals, Inc. Sposoby i kompozycje do modyfikowania docelowego locus
EP3674408A1 (en) 2014-06-16 2020-07-01 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas
MX2016017317A (es) 2014-06-26 2017-08-24 Regeneron Pharma Metodos y composiciones para modificaciones geneticas dirigidas y metodos de uso.
MA40253A (fr) 2014-07-15 2017-05-24 Juno Therapeutics Inc Cellules modifiées pour thérapie cellulaire adoptive
WO2016011428A1 (en) 2014-07-17 2016-01-21 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of treating cells containing fusion genes
US9944933B2 (en) 2014-07-17 2018-04-17 Georgia Tech Research Corporation Aptamer-guided gene targeting
EP3186376B1 (en) 2014-08-27 2019-03-20 Caribou Biosciences, Inc. Methods for increasing cas9-mediated engineering efficiency
EP3450558B1 (en) 2014-08-27 2020-09-30 New England Biolabs, Inc. Synthon formation
US10570418B2 (en) 2014-09-02 2020-02-25 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification
CA2957800A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector immune responses
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016054032A1 (en) 2014-09-29 2016-04-07 The Jackson Laboratory High efficiency, high throughput generation of genetically modified mammals by electroporation
WO2016054326A1 (en) 2014-10-01 2016-04-07 The General Hospital Corporation Methods for increasing efficiency of nuclease-induced homology-directed repair
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2016061073A1 (en) 2014-10-14 2016-04-21 Memorial Sloan-Kettering Cancer Center Composition and method for in vivo engineering of chromosomal rearrangements
US10428310B2 (en) 2014-10-15 2019-10-01 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
CN107027313B (zh) 2014-10-17 2021-09-07 宾州研究基金会 用于多元rna引导的基因组编辑和其它rna技术的方法和组合物
CN107208086A (zh) 2014-10-17 2017-09-26 霍华德休斯医学研究所 基因组探针
WO2016065364A1 (en) 2014-10-24 2016-04-28 Life Technologies Corporation Compositions and methods for enhancing homologous recombination
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
US10883111B2 (en) 2014-11-27 2021-01-05 Danziger Innovations Ltd. Nucleic acid constructs for genome editing
US20170266320A1 (en) 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
EP3230452A1 (en) 2014-12-12 2017-10-18 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016097751A1 (en) 2014-12-18 2016-06-23 The University Of Bath Method of cas9 mediated genome engineering
RU2707137C2 (ru) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации посредством одноэтапного множественного нацеливания
US10190106B2 (en) 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
WO2016112242A1 (en) 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
CN107406842B (zh) 2015-01-09 2021-05-11 生物辐射实验室股份有限公司 检测基因组编辑
WO2016114972A1 (en) 2015-01-12 2016-07-21 The Regents Of The University Of California Heterodimeric cas9 and methods of use thereof
WO2016115326A1 (en) 2015-01-15 2016-07-21 The Board Of Trustees Of The Leland Stanford Junior University Methods for modulating genome editing
ES2955957T3 (es) 2015-01-28 2023-12-11 Caribou Biosciences Inc Polinucleótidos de ADN/ARN híbridos CRISPR y procedimientos de uso
WO2016130697A1 (en) 2015-02-11 2016-08-18 Memorial Sloan Kettering Cancer Center Methods and kits for generating vectors that co-express multiple target molecules
CN107532182A (zh) 2015-02-23 2018-01-02 克里斯珀医疗股份公司 治疗血红蛋白病的材料和方法
WO2016135559A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
US11261466B2 (en) 2015-03-02 2022-03-01 Sinai Health System Homologous recombination factors
GB201504223D0 (en) 2015-03-12 2015-04-29 Genome Res Ltd Biallelic genetic modification
WO2016154579A2 (en) 2015-03-26 2016-09-29 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems

Also Published As

Publication number Publication date
JP6174811B2 (ja) 2017-08-02
JP2016539655A (ja) 2016-12-22
RU2019111616A (ru) 2019-05-06
BR112016013400A2 (pt) 2017-09-26
CA2933433C (en) 2020-11-17
JP6517755B2 (ja) 2019-05-22
US20240052365A1 (en) 2024-02-15
CN110951779B (zh) 2024-04-16
RU2019111616A3 (zh) 2019-11-20
US20150159174A1 (en) 2015-06-11
SG10201700961TA (en) 2017-04-27
US9228208B2 (en) 2016-01-05
US20200291425A1 (en) 2020-09-17
JP2016198110A (ja) 2016-12-01
AU2014360811A1 (en) 2016-07-28
BR112016013400B1 (pt) 2023-02-14
JP6670412B2 (ja) 2020-03-18
AU2019202160A1 (en) 2019-04-18
WO2015088643A1 (en) 2015-06-18
US20150159175A1 (en) 2015-06-11
AU2017210669B2 (en) 2019-01-17
KR101773782B1 (ko) 2017-09-01
MX2021014368A (es) 2022-01-06
AU2017210669A1 (en) 2017-08-24
RU2685914C1 (ru) 2019-04-23
KR20160095150A (ko) 2016-08-10
KR102170502B1 (ko) 2020-10-28
US20160060657A1 (en) 2016-03-03
US10208317B2 (en) 2019-02-19
NZ721985A (en) 2020-10-30
RU2725520C2 (ru) 2020-07-02
US9546384B2 (en) 2017-01-17
US20190112619A1 (en) 2019-04-18
JP2019110925A (ja) 2019-07-11
AU2019202160B2 (en) 2021-06-24
US20170067078A1 (en) 2017-03-09
CA2933433A1 (en) 2015-06-18
CN110951779A (zh) 2020-04-03
CN105980568A (zh) 2016-09-28
KR20170102056A (ko) 2017-09-06
AU2014360811B2 (en) 2017-05-18
RU2016126989A (ru) 2018-01-23
US10711280B2 (en) 2020-07-14
US11820997B2 (en) 2023-11-21
CN105980568B (zh) 2019-12-03
MX2016007654A (es) 2017-08-15
IL245674A0 (en) 2016-06-30

Similar Documents

Publication Publication Date Title
IL277196A (en) Methods and preparations for genome engineering
IL245674B (en) Methods and preparations for the targeted modification of a genome
HRP20211385T8 (hr) Ciljana modifikacija genoma štakora
IL272193B (en) Genome engineering
IL269461B (en) Cytidine-5-carboxamide modified nucleotide preparations and related methods
HK1223401A1 (zh) 用於治療遺傳病狀的方法和組合物
HRP20181632T1 (hr) Postupci i kompoziti za ciljanu modificiranje genoma
EP2981334A4 (en) COMPOSITIONS AND METHODS FOR INHIBITING VIRAL ACTIVITY

Legal Events

Date Code Title Description
FF Patent granted
KB Patent renewed